CLINICAL TRIALS PROFILE FOR HYDROXYUREA

505(b)(2) Clinical Trials for hydroxyurea

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Indication	NCT04247750 ↗	Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)	Recruiting	Azienda Ospedaliero, Universitaria Meyer	Phase 2	2021-01-28	In β-thalassaemia and Sickle Cell Disease (SCD), a significant production of fetal haemoglobin (HbF) may reduce the severity of clinical course and reactivation of γ-globin gene expression in adulthood. HbF induction is one of the best strategies to ameliorate the characteristic symptoms of these diseases. Hydroxyurea (HU) is the only medication, approved by the US Food and Drug Administration, inducing HbF. However, treatments with HU induce sufficient HbF levels in only half of the patients, and side effects including leukopenia and neutropenia are frequently reported. Therefore, novel therapeutic inducers must be identified to develop a personalized treatment in β-thalassaemia and sickle cell anaemia. The availability of new treatments depends on drugs already approved for other indications, and on pharmacokinetics and pharmacovigilance already assessed. Rapamycin (as Sirolimus) is an immunosuppressant agent, approved by the FDA for acute rejection prevention in renal transplant recipients. The ability of this drug to induce γ-globin gene expression in erythroleukemia cell line and erythroid precursors cells (ErPCs) in ß-thalassaemia patients is already known. A clinical investigation on the effects of sirolimus in ß-Thalassaemia aims to evaluate several parameters related to red blood cell status and HbF levels and is a first step for the full clinical development in this new indication.
New Indication	NCT04247750 ↗	Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)	Recruiting	Azienda Ospedaliero, Universitaria Pisana	Phase 2	2021-01-28	In β-thalassaemia and Sickle Cell Disease (SCD), a significant production of fetal haemoglobin (HbF) may reduce the severity of clinical course and reactivation of γ-globin gene expression in adulthood. HbF induction is one of the best strategies to ameliorate the characteristic symptoms of these diseases. Hydroxyurea (HU) is the only medication, approved by the US Food and Drug Administration, inducing HbF. However, treatments with HU induce sufficient HbF levels in only half of the patients, and side effects including leukopenia and neutropenia are frequently reported. Therefore, novel therapeutic inducers must be identified to develop a personalized treatment in β-thalassaemia and sickle cell anaemia. The availability of new treatments depends on drugs already approved for other indications, and on pharmacokinetics and pharmacovigilance already assessed. Rapamycin (as Sirolimus) is an immunosuppressant agent, approved by the FDA for acute rejection prevention in renal transplant recipients. The ability of this drug to induce γ-globin gene expression in erythroleukemia cell line and erythroid precursors cells (ErPCs) in ß-thalassaemia patients is already known. A clinical investigation on the effects of sirolimus in ß-Thalassaemia aims to evaluate several parameters related to red blood cell status and HbF levels and is a first step for the full clinical development in this new indication.
New Indication	NCT04247750 ↗	Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)	Recruiting	Rare Partners srl Impresa Sociale	Phase 2	2021-01-28	In β-thalassaemia and Sickle Cell Disease (SCD), a significant production of fetal haemoglobin (HbF) may reduce the severity of clinical course and reactivation of γ-globin gene expression in adulthood. HbF induction is one of the best strategies to ameliorate the characteristic symptoms of these diseases. Hydroxyurea (HU) is the only medication, approved by the US Food and Drug Administration, inducing HbF. However, treatments with HU induce sufficient HbF levels in only half of the patients, and side effects including leukopenia and neutropenia are frequently reported. Therefore, novel therapeutic inducers must be identified to develop a personalized treatment in β-thalassaemia and sickle cell anaemia. The availability of new treatments depends on drugs already approved for other indications, and on pharmacokinetics and pharmacovigilance already assessed. Rapamycin (as Sirolimus) is an immunosuppressant agent, approved by the FDA for acute rejection prevention in renal transplant recipients. The ability of this drug to induce γ-globin gene expression in erythroleukemia cell line and erythroid precursors cells (ErPCs) in ß-thalassaemia patients is already known. A clinical investigation on the effects of sirolimus in ß-Thalassaemia aims to evaluate several parameters related to red blood cell status and HbF levels and is a first step for the full clinical development in this new indication.

All Clinical Trials for hydroxyurea

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000623 ↗	Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)	Completed	National Heart, Lung, and Blood Institute (NHLBI)		2000-07-01	The purpose of the TCRN is to accelerate research in the management of thalassemia, standardize existing treatments, and evaluate new ones in a network of clinical centers in North America. The emphasis will be on clinical trials that help identify optimal therapy. Therapeutic trials may involve investigational drugs, drugs already approved but not currently used, and drugs currently used.
NCT00000602 ↗	Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	1994-04-01	To determine whether hydroxyurea prevents the onset of chronic end organ damage in young children with sickle cell anemia.
NCT00000586 ↗	Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1992-01-01	To assess the efficacy and safety of orally administered hydroxyurea in the treatment of painful crises in patients with sickle cell anemia.

Clinical Trial Conditions for hydroxyurea

Condition Name

Condition Name for hydroxyurea
Intervention	Trials
Sickle Cell Disease	62
Sickle Cell Anemia	32
Polycythemia Vera	23

Condition MeSH

Condition MeSH for hydroxyurea
Intervention	Trials
Anemia, Sickle Cell	107
Leukemia	36
Leukemia, Myeloid	32

Clinical Trial Locations for hydroxyurea

Trials by Country

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−

Trials by Country for hydroxyurea
Location	Trials
United States	841
Italy	79
Germany	53
Canada	44
France	43

Trials by US State

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−

Trials by US State for hydroxyurea
Location	Trials
Illinois	52
Texas	51
New York	51
North Carolina	44
Maryland	44

Clinical Trial Progress for hydroxyurea

Clinical Trial Phase

Clinical Trial Phase for hydroxyurea
Clinical Trial Phase	Trials
Phase 4	11
Phase 3	55
Phase 2/Phase 3	9

Clinical Trial Status

Clinical Trial Status for hydroxyurea
Clinical Trial Phase	Trials
Completed	140
Recruiting	52
Terminated	33

Clinical Trial Sponsors for hydroxyurea

Sponsor Name

Sponsor Name for hydroxyurea
Sponsor	Trials
National Heart, Lung, and Blood Institute (NHLBI)	29
National Cancer Institute (NCI)	25
Novartis Pharmaceuticals	21

Sponsor Type

Sponsor Type for hydroxyurea
Sponsor	Trials
Other	348
Industry	122
NIH	77

Hydroxyurea: Clinical Trials, Market Analysis, and Projections

Introduction

Hydroxyurea, a non-alkylating antineoplastic agent, has been a cornerstone in the treatment of several critical medical conditions, including sickle cell anemia, certain types of leukemia, and squamous cell carcinomas of the head and neck. Here, we delve into the latest clinical trials, market analysis, and projections for this versatile drug.

Clinical Trials and Recent Findings

Neuroprotective Effects in Sickle Cell Disease

Recent clinical trials have highlighted the potential neuroprotective effects of hydroxyurea in children with sickle cell disease (SCD). The HU Prevent trial, a randomized, double-blind, phase II feasibility/pilot study, investigated the use of dose-escalated hydroxyurea to prevent central nervous system (CNS) injury in children with SCD. The results suggested a significant reduction in the incidence of CNS injury, including silent cerebral infarction, elevated cerebral blood flow velocity, transient ischemic attack, or stroke, in the hydroxyurea group compared to the placebo group[4].

Ongoing Research and Future Directions

These findings support the initiation of a definitive phase III study to further explore the early use of hydroxyurea in infants with SCD. Ongoing research is also focused on expanding the therapeutic uses of hydroxyurea, including its potential in treating other hematological conditions and certain dermatological diseases.

Market Analysis

Current Market Size and Forecast

The hydroxyurea market is experiencing significant growth, driven by several key factors. As of 2023, the market size was valued at USD 1.2 billion and is projected to reach USD 2 billion by 2031, growing at a Compound Annual Growth Rate (CAGR) of 5% from 2024 to 2031[2].

Key Drivers of Market Growth

• Rising Prevalence of Sickle Cell Disease: The increasing global incidence of sickle cell disease is a major driver, as hydroxyurea is a primary treatment for managing this condition.
• Cancer Treatment Applications: Hydroxyurea's use in treating certain cancers, particularly myeloproliferative disorders, is boosting its market demand as cancer rates rise.
• Regulatory Approvals: New indications or formulations of hydroxyurea can expand its market presence.
• Awareness and Education: Increased awareness among healthcare providers and patients about the benefits of hydroxyurea is leading to higher adoption rates.
• Generic Availability: The presence of generic versions makes hydroxyurea more accessible, driving up usage in various patient populations.
• Research and Development: Ongoing clinical trials and research into additional therapeutic uses are expected to enhance market growth[2].

Market Segmentation

The global hydroxyurea market is segmented based on indication, formulation, end-user, and geography.

• Indication: Hydroxyurea is primarily used for sickle cell anemia, chronic myeloid leukemia, and squamous cell carcinomas of the head and neck.
• Formulation: The market includes various formulations such as capsules, oral solutions, and others.
• End-User: Hospitals, clinics, home care settings, and research institutions are the main end-users, with hospitals handling the most complex cases[2].
• Geography: North America, particularly the United States, holds a prominent position due to high disease prevalence and advanced healthcare infrastructure. Europe and the Asia-Pacific region are also significant markets, driven by growing healthcare needs and investments[2].

Market Projections

Sickle Cell Disease Treatment Market

The hydroxyurea segment dominates the sickle cell disease treatment market, which is expected to grow significantly. The global sickle cell disease treatment market is projected to be worth around USD 6.9 billion by 2032, growing at a CAGR of 14.10% from 2024 to 2032. Hydroxyurea's therapeutic benefits and its widespread use in managing sickle cell anemia are key factors driving this growth[3].

Competitive Landscape

Major players in the hydroxyurea market include Bristol-Myers Squibb Company, Teva Pharmaceutical Industries Ltd., Novartis AG, Pfizer Inc., Mylan N.V., Hikma Pharmaceuticals PLC, Amgen Inc., Merck & Co., Inc., Apotex Inc., and Fresenius Kabi AG. These companies are involved in various strategies such as expanding product portfolios, enhancing distribution networks, and investing in research and development to maintain their market positions[2].

Economic and Healthcare Infrastructure Impact

Cost-Effectiveness

Hydroxyurea's cost-effectiveness compared to other treatments is a significant driver, especially in resource-limited settings. This makes it an attractive option for healthcare systems looking to manage chronic and complex conditions efficiently[2].

Regional Dynamics

The market is influenced by regional healthcare systems, regulatory environments, and disease prevalence. North America and Europe are leading markets due to their advanced healthcare infrastructure and high incidence of relevant diseases. The Asia-Pacific region is emerging as a lucrative market, driven by increasing healthcare access and government funding for healthcare initiatives[2].

Key Takeaways

• Clinical Trials: Recent trials suggest hydroxyurea has neuroprotective effects in children with sickle cell disease, supporting further research.
• Market Growth: The hydroxyurea market is projected to grow at a CAGR of 5% from 2024 to 2031, driven by increasing disease prevalence and new therapeutic applications.
• Market Segmentation: The market is segmented by indication, formulation, end-user, and geography, with hospitals being the primary end-users.
• Competitive Landscape: Major pharmaceutical companies are key players, with strategies focused on product expansion and research.
• Economic Impact: Hydroxyurea's cost-effectiveness and regional healthcare dynamics play crucial roles in its market growth.

FAQs

What are the primary indications for hydroxyurea?

Hydroxyurea is primarily indicated to reduce the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia, as well as for treating certain types of leukemia and squamous cell carcinomas of the head and neck[1].

What are the potential side effects of hydroxyurea?

Hydroxyurea can cause serious side effects, including secondary cancer, birth defects, testicular atrophy, and decreased spermatogenesis. It is also mutagenic and clastogenic in various cell types[1].

How is hydroxyurea administered?

Hydroxyurea is administered orally, with peak plasma concentrations reached within 2 hours. It has nearly complete bioavailability in cancer patients and those with sickle cell syndrome[1].

What is the current market size and forecast for hydroxyurea?

The hydroxyurea market was valued at USD 1.2 billion in 2023 and is projected to reach USD 2 billion by 2031, growing at a CAGR of 5% from 2024 to 2031[2].

Which regions are leading the hydroxyurea market?

North America, particularly the United States, holds a prominent position due to high disease prevalence and advanced healthcare infrastructure. Europe and the Asia-Pacific region are also significant markets[2].

What are the key drivers of the hydroxyurea market growth?

Key drivers include the rising prevalence of sickle cell disease, cancer treatment applications, regulatory approvals, increased awareness, generic availability, and ongoing research and development[2].

Sources

1. DrugBank: Hydroxyurea: Uses, Interactions, Mechanism of Action.
2. Verified Market Research: Hydroxyurea Market Trends, Size, & Forecast.
3. Market.US: Sickle Cell Disease Treatment Market Size | CAGR of 14.10%.
4. PubMed: Hydroxyurea to prevent brain injury in children with sickle cell disease.
5. Market Research Reports: Global Hydroxyurea Market 2024 by Manufacturers, Regions, Type and Application, Forecast to 2030.